Our work describing the development of a potent epigenetic editor targeting human #PCSK9 for durable reduction of LDL-C is out in @NatureMedicine today!
Cc: @chromamedicine@nchromabio
1/9
https://t.co/cnelNtMT0W
Amazing news for chronic HBV patients: bepirovirsen B-Well Ph3 published in @NEJM & presented at #EASLCongress: 19% functional cure (HBsAg loss + undetectable DNA ≥24 wks post-treatment) vs 0% placebo. This is the new bar. Congrats to @GSK@ionispharma https://t.co/jSd2SZZ4V8
Happy birthday to two of my favorite people in the world - Ralph DeBerardinis (@rjdlab), who turns 50 today, and Alan Hall, who would have been 68. In addition to sharing a birthday, Ralph and Alan have quite a bit in common (thread)
https://t.co/zTnJTOzbBk
Thanks for sharing view, @jrkelly!
This is a polarizing issue, as you know.
I, for one, do not support breaking access to China-based manufacturing or innovation. It would harm patients, and that MUST remain our Northstar.
On the other hand, I think China’s recent advances must be a “Sputnik moment” for the U.S.. For starters, we should stop and reverse the self-inflicted damage created by certain Administration policies, including reduced NIH funding, FDA instability, drug price controls, H1B visa restrictions, and an anti-Vax/anti-science agenda.
Then, we should find smart ways to increase our global competitiveness, including reducing barriers to efficient clinical evidence generation, creating incentives for U.S. clinical trial and manufacturing, and reforming PBMs/340B to ensure reward for the innovator, not the middleman.
We can win this competitive race with American ingenuity and resolve, not with walls and barriers!
Very good news from @REGENXBIO and for Duchenne patients
Presentation tomorrow @ASGCTherapy#ASGCT2026
Regenxbio says Duchenne gene therapy succeeded in clinical trial, paving way for FDA submission https://t.co/rmjWjxWM64 via @statnews
🚨 Excited to share a new paper in Cell! Human genetics led us to HOTSCRAMBL, a HOXA-locus lncRNA that regulates 🩸#stemcell self-renewal and HOXA9 splicing, with implications for AML.
Amazing work by @lvchosen1 with @silvirouskin, @Armstrong_dfci, and many more!
https://t.co/Kralk3tmzg
🌟From the desk of @SJMorrison_: It's a great day for CRI, which now has 2 @theNASciences members. Congratulations to Ralph DeBerardinis, M.D., Ph.D., and his laboratory on this recognition for the quality and impact of their work! #relentlessdiscovery
https://t.co/drqKPSLL3z
👁🗨We turn science into sight.
Congratulations to our Director @IOB_Roska on receiving the 2026 Future Vision Award alongside Connie Cepko & Daniel Ting. A recognition of long-term research now reaching patients.
🎬Watch the short movies: https://t.co/mbWgbGdpbN
#Vision #Retina #Ophthalmology #Blindness #Neuroscience #ScienceInnovation #VisionRestoration #EyeHealth #BiomedicalResearch
Congrats to all involved in getting this important medicine to patients - teams @Regeneron & DecibelTx, Joe Burns, Adam Palermo, Jonathan Whitton, @jkeilty, Laurence Reid, many others…
& big props to @Regeneron for providing Otarmeni for free in the US
https://t.co/Beon2jHvlt
“We are losing scientists, we’re losing our IP, we’re losing physicians, we’re losing the best researchers, and we’re going to lose our biosecurity,” Kennedy said.
Well, maybe we should STOP all the self-inflicted damage by: funding academic research, attracting best scientists from all over the world with H1B visa program, supporting a strong, consistent FDA without political influence, supporting our public health infrastructure, and supporting scientists!
Congratulations to Jean Bennett, Katherine A. High and Albert Maguire, winners of the 2026 Breakthrough Prize in Life Sciences, for developing a therapy for inherited retinal degeneration that became the first FDA-approved gene therapy for a genetic disease. https://t.co/awmRtAX5vT
@Penn@ChildrensPhila@RockefellerUniv
That Phase 3 overall survival data is in second line #PancreaticCancer which makes the numbers even more astounding. I cannot recall any other example in pancreatic cancer when OS in a Phase 3 trial basically doubled over existing standard of care.
https://t.co/89kVHEmSMh
Finally, best news of the morning (and something you didn't expect so soon)...
Revolution Medicines $RVMD daraxonrasib Ph3 results in second-line pancreatic cancer.
Median overall survival in ITT patients (KRAS mutants + wild type combined) Dara 13.2 months vs chemo 6.7 months.
OS Hazard ratio 0.40
A decade ago, these drugs tore apart the FDA. Today, they might be some patients’ best hope
Another wonderful piece from @Jasonmmast
https://t.co/zOvLrdAPKi via @statnews
Resharing this piece I wrote last year on human genetics and drug discovery.
India’s genetic diversity is an underexplored asset, one that can fundamentally reshape how we discover new medicines.
This is exactly the direction we’re building towards 🇮🇳
https://t.co/0yh2gmaUaP
Humans with function-disrupting variants in the myostatin gene (MSTN) have increased skeletal muscle mass and strength, and less adiposity
https://t.co/bl25n6ijJv